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NON-CONFIDENTIAL PRESENTATION November 2013 Founded February 2009, - PowerPoint PPT Presentation

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NON-CONFIDENTIAL PRESENTATION November 2013 Founded February 2009, based in Vancouver, British Columbia Formerly AlCana Technologies Inc Highly experienced team developing lipid nanoparticle delivery systems Facilities for chemistry,

  1. NON-CONFIDENTIAL PRESENTATION November 2013

  2. Founded February 2009, based in Vancouver, British Columbia Formerly AlCana Technologies Inc Highly experienced team developing lipid nanoparticle delivery systems Facilities for chemistry, formulation and preclinical studies with access to additional resources at the University of British Columbia (UBC) Nov 2013 2

  3. Systemic lipid nanoparticles for intracellular delivery of molecular therapeutics – primarily nucleic acids Pharmaceutical applications: Protein replacement therapeutics (mRNA or plasmid delivery) Protein knockdown therapeutics (antisense) Nov 2013 3

  4. Multi-component carrier Small, uniform sized particles (~80 nm) Low surface charge in blood compartment Nov 2013 4

  5. Proof of Concept Studies using reporter mRNA Luciferase mRNA encapsulated in LNP – encapsulation efficiency >90%. In vivo administration (i.v.) to mice followed by: Quantitative luciferase expression determined ex vivo (Steady-Glo Luciferase assay kit: Promega) Live imaging (IVIS system) Nov 2013 5

  6. DOSE RESPONSIVE PROTEIN EXPRESSION Efficient mRNA delivery to liver. Luciferase expression proportional to mRNA dose (0.1-3.0 mg/kg) Nov 2013 6

  7. COMPARATIVE LNP POTENCY Acuitas LNP vs Phua et al. LNP 1 Luciferase expression in liver 4 hours after iv administration 1 Phua, Leong and Nair, J. Control Release, 166(3) 227-233, 2013 Nov 2013 7

  8. Focus on partnerships in protein replacement therapeutics Acuitas supports: Preclinical screening to select Lead Products Protection of Lead Products through new and existing IP rights Analytical, biophysical and preclinical characterization of Lead Products Transition from preclinical to clinical development Nov 2013 8

  9. Corporate Contact: Dr. Thomas Madden, President & CEO Phone: 604-880-6157 Email: tmadden@acuitastx.com Scientific Contact: Dr. Michael Hope, CSO Phone: 604-837-9239 Email: mhope@acuitastx.com Website: www.acuitastx.com LNP Technology developed under limited license from Tekmira Pharmaceuticals Corporation Nov 2013 9

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